What is the appropriate stem cell transduction assay?
Hematopoietic stem cells have been targeted for genetic modification for over 2 decades. In the 1980s it was shown that mouse hematopoietic stem cells could be genetically modified with a degree of efficiency predicted to be therapeutic for many human diseases8,9. These results prompted a high level of enthusiasm until it became apparent that the transducibility of hematopoietic stem cells is just another feature to distinguish mice from men.Because stem cell transduction protocols developed in the mouse did not translate into efficient gene transfer protocols in human studies, many investigators started to study stem cell transduction in dogs10,11,12,13,14 and nonhuman primates15,16,17,18,19,20,21. Similar to the initial clinical trials, these early studies in large animal models yielded disappointingly low gene transfer rates. These initial difficulties in achieving high-level stem cell transduction demonstrated that results from large animal models were much more predictive of human
